异基因造血干细胞移植治疗成人FLT3-ITD阳性急性髓系白血病40例远期疗效分析

OBJECTIVE: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of acute myeloid leukemia (AML) patients with FLT3-ITD mutation. METHODS: From September 2008 to December 2016, 40 AML patients with FLT3-ITD mutation were enrolled in the study. The therapeutic process, outcomes and prognostic factors were retrospectively analyzed. RESULTS: The median of WBC at initial diagnosis was 35.0 (range 1.7–185.0) ×10(9)/L. The median course number of chemotherapy was 4 (range 2–7). At the time of transplantation, 34 patients were at the first complete remission (CR(1)) stage, and the other 6 ones were non-remission after chemotherapy. 24 patients received allogeneic transplants from an HLA-matched sibling donor, 7 cases from a HLA-matched unrelated donor, the remaining 9 ones received allograft from a haploidentical donor. The rate of 3-year overall survival (OS) and disease free survival (DFS) in all patients were both 74.3% (95% CI 60.4%–88.2%). The 3-year cumulative incidences of disease relapse and non-relapse mortality were 7.5% (95%CI 1.9%–18.4%) and 18.2% (95% CI 7.9%–32.0%), respectively. More than one course of chemotherapy before achieving CR(1) and the occurrence of acute GVHD after transplantation were associated with poor outcome in terms of OS and DFS. The relapse rates were significantly lower in patients receiving transplantation at CR(1) stage [0 vs 50.0% (95%CI 77.7%–82.9%), P<0.001] and achieving CR(1) after one course induction therapy [0 vs 16.7% (95%CI 3.9%–37.3%), P=0.020]. CONCLUSION: Allo-HSCT was an efficient approach for AML patients with FLT3-ITD mutation. Patients obtained better survival, especially for those achieving CR after one course induction therapy and receiving transplantation at CR(1) stage.